Davunetide for PSP on Fast Track with FDA

July 28, 2011

Allon Therapeutics Inc. have issued an update on the progress of the pivotal Phase 2/3 clinical trial that is evaluating the Company’s lead neuroprotective drug candidate davunetide as a potential treatment for progressive supranuclear palsy (PSP).

PSP and FTD

PSP is one of a group of progressive neurodegenerative disorders called frontotemporal dementias (FTD) that affect movement, speech, and behavior, and for which there are no approved treatments. Approximately 25,000 and 50,000 persons, in the U.S. and EU respectively, have PSP. Approximately half of FTDs, including PSP, are tauopathies, or involve impairment of the tau protein in brain cells. Allon expects that demonstrating efficacy in PSP will define the opportunity to use davunetide in other tau-related diseases, such as Alzheimer’s, schizophrenia, Parkinson’s and several subtypes of FTD.

Davunetide for PSP

The pathology of PSP and Alzheimer’s disease is similar in that both diseases involve impairment of the brain protein tau – and davunetide is the most advanced tau therapy in the world. Allon has demonstrated a strong scientific and clinical rationale for the potential efficacy of davunetide in PSP, driven by the demonstration of activity in preclinical models of tauopathies and clinical efficacy in amnestic mild cognitive impairment, an early form of Alzheimer’s disease known to be associated with the build-up of tau tangles. Davunetide has received orphan drug designation in the United States and the European Union as a treatment for PSP, and fast track status from the FDA.

Drug Trials

Gordon McCauley, Allon’s President and CEO, said the trial has enrolled approximately 50% of the 300 patients specified in the protocol. Enrollment began in the fourth quarter of 2010. The trial is being conducted under a Special Protocol Assessment (SPA) granted by the United States Food and Drug Administration (FDA), which ensures that the agreed clinical trial design meets the FDA’s expectations for a pivotal study.

“Our progress is right on track with our estimate to complete enrollment by the end of 2011 and to report data about a year later,” said McCauley. “The pace of enrollment is a testament to the patients, caregivers, investigators, and advocacy groups who have been tremendous partners, and who certainly hope this trial will generate data necessary for marketing approval of davunetide as a treatment for PSP.”

McCauley also said the trial’s Data Safety and Monitoring Board (DSMB) recently approved the continuation of the trial. A DSMB is an independent group of clinical experts with the primary responsibility of monitoring the safety and well-being of subjects and to assure scientific integrity of the study. A DSMB is independent of the company and the clinical investigators, who are blinded from the safety and efficacy data until all treatment has been completed.

This multi-national study is being conducted at premier medical institutions in the United States, Canada, the United Kingdom, France, Germany, and Australia. A list of the clinical trial sites can be found at www.clinicaltrials.gov.

Citation

Allon Therapeutics Inc. is a clinical-stage biotechnology company focused on bringing to market innovative central nervous system therapies. Allon’s lead drug davunetide is proceeding in a pivotal Phase 2/3 clinical trial in an orphan indication, progressive supranuclear palsy (PSP), under an SPA with the FDA. This pivotal trial is based upon statistically significant human efficacy demonstrated in amnestic mild cognitive impairment (a precursor to Alzheimer’s disease), cognitive impairment associated with schizophrenia, and positive biomarker data.

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